This guidance is intended for stakeholders who intend to enroll pregnant women or to retain women on trial who become pregnant during the course of the trial.

The guidance encourages further discussion among stakeholders regarding scientific and ethical considerations for including pregnant women in clinical trials.

Clinical trials on pregnant women introduce complex issues due to risk-benefit considerations for both the pregnant woman and fetus and the reliance of the fetus on the mother.

Careful consideration of potential risks to the fetus should be analyzed prior to designing trials that include pregnant women.

The focus of the guidance is both on therapies that are intended to treat acute conditions that occur during pregnancy and for common conditions that may affect women of childbearing age.

The focus is notably on treatments for chronic conditions the pregnant woman may already experience prior to pregnancy and additional information about risks and benefits to the pregnant woman and the fetus of continuing therapy.

Background:

Limited data on safety and risk-benefit profile are available for pregnant women until after drug approval.

Clinical trials are not typically conducted on pregnant women.

Drug labeling for safety during pregnancy is typically based on animal laboratory data and limited data is available on risks and benefits for use in pregnant women.

Lack of information on risks and benefits of drug use in pregnancy may lead to undertreated medical conditions, which may lead to negative outcomes for the woman and/or fetus.

When drugs are chosen to treat conditions during pregnancy, limited data on safety is available, which impairs informed decision making.

Data collected on safety of drug use in pregnant women is commonly accomplished with marketed products.

Pregnant women have typically been avoided in clinical trials due to the potential risk to the fetus.

There are potential benefits to pregnant women being included in clinical trials. For instance, 1) if a treatment is not otherwise available, 2) to compare standard of care therapies for conditions related to pregnancy, 3) a serious condition.

Benefits for including pregnant women in trials include:

Establish which drugs are safe and effective to use during pregnancy.

Potential for direct benefit to the woman and/or fetus.

Access to treatments that may not otherwise be available.

Establishment of a safe and effective dose during pregnancy.

Physiological differences may occur during pregnancy that alter the metabolism, absorption, distribution, and/or excretion of drugs.

Dosing schemes established on non-pregnant subjects may not offer the best comparison.

Lack of knowledge of what constitutes safe and effective drug use in pregnant women is a public health concern that the FDA begins to address in this guidance. Join us in Part 2 of “FDA Drafts Guidance for Consideration for Inclusion of Pregnant Women in Clinical Trials” to learn about ethical considerations that need to be addressed prior to including pregnant women in clinical trials.